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PREPL deficiency with or without cystinuria causes a novel myasthenic syndrome

OBJECTIVE: To investigate the genetic and physiologic basis of the neuromuscular symptoms of hypotonia-cystinuria syndrome (HCS) and isolated PREPL deficiency, and their response to therapy. METHODS: We performed molecular genetic, histochemical, immunoblot, and ultrastructural studies, investigated...

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Détails bibliographiques
Auteurs principaux: Régal, Luc, Shen, Xin-Ming, Selcen, Duygu, Verhille, Chantal, Meulemans, Sandra, Creemers, John W.M., Engel, Andrew G.
Format: Artigo
Langue:Inglês
Publié: Lippincott Williams & Wilkins 2014
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4001208/
https://ncbi.nlm.nih.gov/pubmed/24610330
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000000295
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